Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06421636 | A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body | PHASE2 | RECRUITING | 95 | — | — | Sep 30, 2024 | Jan 24, 2028 | Mar 18, 2026 | 26 | United States, Georgia +7 |
| Arm | Type | Description |
|---|---|---|
| Part A High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| Part B High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| Name | Type | Description |
|---|---|---|
| REGN7999 | DRUG | Administered subcutaneous (SC) |
| Placebo | DRUG | Administered SC |
Key Inclusion Criteria: 1. Clinical diagnosis of NTDT as described in the protocol 2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening 3. Serum ferritin ≥ 300 ng/mL as described in the protocol Key Exclusion Criteria: 1. Hemoglobin ≤ 8 g/dL at screening 2. Any RBC transfusi...